Clin Res Cardiol (2023). https://doi.org/10.1007/s00392-023-02180-w
Tafamidis in cardiac wt-ATTR-amyloidosis – one year follow-up data
I. Yan1, J. C. M. Franke1, R. Frauen2, N. Diermann1, C. Chevalier1, P. Kirchhof1, K. Müllerleile3, M. Patten-Hamel1
1Klinik für Kardiologie, Universitäres Herz- und Gefäßzentrum Hamburg, Hamburg; 2Kardiologie, Universitätsklinikum Hamburg-Eppendorf, Hamburg; 3Kardiologische Praxis Orchideenstieg, Hamburg;

Aim

Cardiac Wild-type transthyretin amyloidosis (wtATTR) is a rapidly progressive disease mainly affecting elderly males and which is associated with high mortality rates in the absence of targeted therapy. Since 2020 Tafamidis is available as first-line therapy in patients with cardiac ATTR amyloidosis, acting as a chaperone, that selectively stabilizes TTR and prevents tetramer dissociation and amyloid formation. So far, long-term effects on cardiac function and remodeling are poorly understood. In this single center study the one-year effect of Tafamidis on clinical, laboratory and echocardiographic parameters in patients with wtATTR were examined.

Methods and Results 

A total of 43 patients diagnosed with wtATTR and referred to our university outpatient clinic between November 2020 and August 2022 were treated with Tafamidis 61 mg/d. Clinical examination, 6-min walk test, cardiac markers, ECG and transthoracic echocardiography were performed at baseline and after 6 and 12 months of treatment. 

The mean age was 80 ± 8,5 yrs, 7% (n=3) were females. The clinical presentation was divided in NYHA classification: NYHA I (n=10), NYHA II (n=20), NYHA III (n=13). During the follow-up 5 patients died, 4 of them were NYHA III at baseline and 2 of them had a reduced left ventricular ejection fraction of 35%.

In accordance with the approval study, cardiac markers, such as NT-proBNP, hscTnIand 6-min walk test results (344,2m ±88,1m vs. 362,6m ±89,3m vs. 345,7m ±90,7m p= 0,16), remained stable 12 months under therapy with Tafamidis. Besides, echocardiographic parameters, such as left ventricular ejection fraction (LVEF), diastolic dysfunction (E/A, E´), left ventricular wall thickness (IVSd, PWd), left atrial size (LA), global longitudinal strain (GLS) and TAPSE remain also stable from baseline to one year follow-up.

Conclusion

In this single center observational study, patients with cardiac wtATTR showed stable clinical, laboratory and cardiac imaging parameters one-year after therapy with Tafamidis in this otherwise progressive disease. 

 

 

Echocardiography

Baseline

6 months

12 months

p

Ejection fraction (%)

50,5 ± 11,5

52,1 ± 9,9

51,0 ± 8,0

0,73

IVSd (mm)

18,5 ± 3,0

19,5 ± 2,3

20,4 ± 4,1

0,12

PWd (mm)

15,8 ± 3,1

17,0 ± 2,3

16,5 ± 2,0

0,21

LA (ml) 

81,4 ± 24,7

78,7 ± 21,0

81,4 ± 18,1

0,99

TAPSE (mm)

16,9 ± 4,3

16,6 ± 3,9

16,4 ± 3,7

0,74

RVP (mmHg)

31,5 ± 10,1

34,1 ± 12,6

37,9 ± 14,1

0,20

GLS 

-10,47 ± 3,2

-10,17 ± 4,6

-11,43 ± 4,6

0,75

E

87,9 ± 21,3

92,1 ± 28,6

99,0 ± 30,9

0,19

A

56,0 ± 39,7

54,1 ± 29,7

49,3 ± 30,9

0,61

E/é

17,5 ± 6,9

19,3 ± 7,9

19,7 ± 8,0

0,27

E/A

2,25 ± 1,3

2,15 ± 1,0

2,59 ± 1,5

0,42

 

Table 1 Echocardiographic parameters 

Data are shown in mean ± SD. 

 

  
https://dgk.org/kongress_programme/jt2023/aP520.html